Mr. Vowels et al., USE OF GRANULOCYTE-MACROPHAGE COLONY-STIMULATING FACTOR IN 2 CHILDRENTREATED WITH CORD-BLOOD TRANSPLANTATION, Blood cells, 20(2-3), 1994, pp. 249-255
Cord blood contains stem cells in amounts similar to or slightly less
than those present in a bone marrow collection to be used for bone mar
row transplantation (BMT), Too few cord blood transplants (CBT) have y
et been performed to define the ability to achieve engraftment and the
rate of engraftment, Two cord blood transplants have been performed u
sing granulocyte-macrophage colony stimulating factor (GM-CSF) to hast
en engraftment, Two children, aged 5 and 6 years received a CBT using
HLA-identical stem cells collected at the birth of a sibling, One chil
d had X-linked lymphoproliferative disease (XLP), and the other, acute
lymphoblastic leukemia in second complete remission, One had an ABO a
nd one an Rh blood group mismatch, Conditioning therapy consisted of c
yclophosphamide, melphalan, and antithymocyte globulin or busulphan an
d cyclophosphamide, Graft-versus-host disease prophylaxis was methotre
xate and cyclosporine or cyclosporine, Both children were given GM-CSF
at 5 mu g/kg/day from day 1 until the absolute neutrophil count (ANC)
reached 1.0 x 10(9)/L for 3 consecutive days, If this level was not r
eached by day 14, the dose of GM-CSF was doubled, Both children engraf
ted rapidly, with ANCs reaching 0.5 x 10(9)/L in 12 and 16 days, Engra
ftment was confirmed by blood group in both and sex chromosome typing
in one, Both children developed mild GVHD localized to skin, which res
olved with steroid therapy, The child with XLP was cured and has survi
ved for 34 months; the second child has survived 27 months with normal
marrow function but has had a relapse of leukemia, CBT has been succe
ssfully undertaken in young children, Myeloid engraftment occurred at
the same rate as that seen in children receiving a BMT followed by GM-
CSF.